An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

Phase 3RecruitingNCT06700096

Biocad24 enrolled

Overview

The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity ≤2% and without FIX inhibitor. The study will have an open-label single-arm design.

After signing the ICF and screening examination the subjects are to be included in the Non-interventional lead-in period in which the subject will receive standard FIX prevention. The lead-in period will last for at least 6 month for every subject. After completiong the lead-in period subjects will enter the main (interventional) period. At the first visit of the main period subjects will recieve investigational product ANB-002. The main period ends 18 months after the administration of ANB-002, after which subjects will switch to the follow-up period and will be evaluated up to 5 years after the ANB-002 infusion.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemophilia B

Interventions

ANB-002GENETIC

Adeno-associated viral vector carrying the FIX gene single infusion

Eligibility

Sex: MALEMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Men diagnosed with hemophilia B aged 18 or older * FIX activity ≤2% * Absense of FIX inhibitor * ≥150 previous exposure days of treatment with FIX concentrates Exclusion Criteria: * Any diseases of blood and hematopoietic organs other than hemophilia B * A history of any gene therapy, including ANB-002 * Diagnosed HIV-infection, not controlled with anti-viral therapy * Active HBV or HCV infection * Anti-AAV5 antibodies * Any active systemic infections or recurrent infections requiring systemic therapy * Any other disorders associated with severe immunodeficiency * Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder * Malignancies with less than 5 years of remission

Locations (14)

Chelyabinsk Regional Clinical Hospital

Chelyabinsk, Russia

Outcomes

Primary Outcomes

Annualized bleeding rate (ABR) before and after a single dose of ANB-002

A comparison will be made between the mean ABR values in the lead-in period and within 12 months after stabilization of endogenous FIX activity, but not later than 18 months after the ANB-002 dose

Time frame: Lead-in period: at least 6 months prior to ANB-002 infusion (Lead-in arm); Month 12 to Month 18 post infusion (ANB-002 arm)